How The Compounding Scandal Benefits One Drugmaker

One company's pain is another company's gain. For the past few years, a significant number of ophthalmologists have used the Avastin cancer medication to treat age-related macular generation, a common afflication among the elderly, even though the drug was never approved for this use. Avastin gained popularity because, until recently, the only alternative was another Roche drug called Lucentis, which is about 40 times more expensive for each injection.

However, the off-label use of Avastin prompted concerns - particularly from Roche, which wanted the bigger sales associated with the higher-priced Lucentis - because rejiggering Avastin into vials was linked to reports of infections and blindness. At one point, the US Department of Veteran Affairs suspended Avastin usage for treating wet AMD (back story). But the landscape changed a year ago when the FDA approved yet another drug for wet AMD, Eylea, which is sold by Regeneron Pharmaceuticals.

In the recently ended third quarter, Eylea captured roughly one-third of the branded market share for wet AMD, according to Regeneron ceo Leonard Schleifer. Moreover, the branded market is growing, he believes, because there were additional indications for Lucentis for treating diabetic macular edema, the patient population expanded due to age and more patients switched from Avastin. "The market dynamics are not stagnant," he said at the recent Credit Suisse Healthcare Conference (see this).

Yet another dynamic is shifting quickly. Thanks to medicines made by the New England Compounding Center, which led to an outbreak of fungal meningitis that has claimed 33 lives (look here), there is increased betting that Eylea will capture still more market share, since compounded Avastin is used by ophthalmologists to treat wet AMD. "I think there will be regulation," Schleifer predicted. "And... anything that would make Avastin safer would be good for our patients and our doctors."

Indeed, the NECC scandal has been cited as an example in which compounded Avastin may be worrisome. In 2006, the FDA sent a warning letter to the compounder that cited, among other things, concerns about marketing anesthetic creams and potential microbial contamination associated with splitting and repackaging Avastin. The agency also tagged NECC for sending promotional materials to ophthalmologists, when the drug was not approved for wet AMD (see here).

The recent congressional hearings held by House and Senate committees are being interpreted as a good indication that bi-partisan support can be mustered to pass legislation that would strengthen FDA oversight of compounders. Meanwhile, the American Society of Retinal Specialists sent an e-mail alert to inform its members that 15 states are now requiring an individual patient script for every compounded medication order, according to Deutsche Bank analyst Robyn Karnauskas.

Given recent events, it would appear likely that Avastin, which is typically made in bulk quantities, would be included on such a list and more states will adopt the same policy, especially after the FDA and state health and pharmacy officials hold a scheduled meeting on December 19 to discuss compounding. "If this is truly implemented," she writes in an investor note, "we think that this could restrict Avastin availability."

All of which, of course, bodes well for Regeneron. And with a market for wet AMD treatments reaching $1.5 billion last year and continuing to grow, the little drugmaker is poised to turn Eylea into a blockbuster. This helps explain why Regeneron shares moved roughly 10 percent over just a few days in mid-November - Schleifer made his comments at the same time that Congressional hearings were getting under way to examine the compounding controversy.

"We believe Congress is, ultimately, poised to regular comounding pharmacies more over time," Karnauskas wrote, and that "initial federal and state (government) activity could lead to lessened Avastin demand in the near term, before legislation could occur." Mix the negative headlines with stricter state requirements and you have a more potent cocktail than anything a compounder could grind. And who benefits? Anyone selling Lucentis and... Eylea.